OBJECTIVE To analyze the safety and effectiveness of abatacept (ABA) given in routine care to patients with systemic sclerosis (SSc). METHODS Retrospective multicenter observational study that enrolled patients with SSc treated with ABA. We collected epidemiological data and clinical outcomes. First, we analyzed the frequency of adverse effects. Secondly, we compared the evolution of different organ manifestations during ABA treatment. We collected data from 6 months before start of therapy to the last follow-up the following parameters modified Rodnan Skin Score (mRSS), joints, lung and gastrointestinal involvement, concomitant medications, and laboratory tests. RESULTS Data on twenty-seven patients with SSc were collected (93% females; 67% limited SSc). Rheumatoid arthritis was the most frequent concomitant autoimmune disease. ILD was present in 15 patients. Anti-Scl 70 antibodies were present in 13 patients and rheumatoid factor and ACPA antibodies were present in eight and seven patients respectively. The main indication to use abatacept was joint involvement (59%) followed by myositis (26%). A total of 16 adverse effects were reported in 28 months of abatacept treatment including five that required hospitalization. Most of them occurred in the first 3 months after starting abatacept. https://www.selleckchem.com/products/jnj-64264681.html After 12 months, the number of tender and swollen joints decreased compared to baseline (p less then 0.03 and p less then 0.02 respectively). Moreover, a beneficial effect of abatacept on HAQ-DI at 3 and 6 months (p less then 0.05) and on morning stiffness at 6 and 12 months (p less then 0.03) was observed. We also observed a decrease in the modified Rodnan skin score (p less then 0.05). No changes in lung or gastrointestinal involvement were found. CONCLUSIONS ABA demonstrated a good safety profile and seems to have some effectiveness on joint involvement and related disability in SSc patients treated in routine care. BACKGROUND Information about the possible effect of rheumatic diseases on male sexual function and reproduction (sexual health) is scarce and difficult to summarize. Factors known to impair sexual health, such as inflammation, medication use and hypogonadism can be present in a significant proportion of male patients with rheumatic diseases. OBJECTIVES The objective of our study was to systematically review the literature for the influence of paternal rheumatic disease on sexual health, such as sexual function, reproductive hormones, male fertility, pregnancy and offspring outcomes. DATA SOURCES English language articles identified through Embase, MEDLINE, Cochrane Central Register of Controlled Trials (CENTRAL), Web of Science, Google Scholar and the Clinical trial registries of Europe and the USA published until February 2019. STUDY APPRAISAL AND SYNTHESIS METHODS Literature was synthesized in narrative form and in summary tables. Outcomes were categorized as sexual function, reproductive hormones, fertilithis association and discuss it with their patients. IMPLICATIONS OF KEY FINDINGS Sexual health of men with rheumatic diseases can be impaired by the disease itself. Especially in men trying to conceive, information on sexual function, reproductive hormones and sperm quality are needed to identify these problems. Treatment resulting in lower disease activity can improve overall sexual health in man with rheumatic diseases and facilitate their journey to fatherhood. SYSTEMATIC REVIEW REGISTRATION NUMBER PROSPERO 2018 CRD42018099845. BACKGROUND Rett syndrome is a neurodevelopmental disorder with potential for improvement through novel targeted therapeutics. Reliable outcome measures are critical to the development of treatments. We examined the merits and flaws of the Rett Syndrome Behavioural Questionnaire, an outcome measure for clinical trials. METHODS The Rett Syndrome Behavioural Questionnaire was administered alongside other clinical scales in three cohorts, an online survey, a clinic-based study, and the screening period for a clinical trial. Data were collected from individuals with Rett syndrome and related disorders at three time points, separated by a minimum of one&nbsp;week and a maximum of two&nbsp;months. We hypothesized that for clinical trial use, little change should occur among visits. Distribution statistics, internal consistency, intraclass correlation coefficient, percent agreement, and Cohen's kappa were examined. RESULTS Among 149 with classic Rett syndrome, the Rett Syndrome Behavioural Questionnaire was completed 377 times. Median total score was 33, ranging from 3 to 73. Of the 51 items tested in the original Rett Syndrome Behavioural Questionnaire study, 24 exhibited either floor or ceiling effects. Friedman's analysis of variance revealed significant difference among visits (P&nbsp;=&nbsp;0.024), and graphical analysis using Bland-Altman plots demonstrated systematic positive bias with a 95% confidence interval including up to 12.9 points higher to 15.7 points lower at retest. Median agreement measured by kappa was 0.53 for retest at visit 2 and 0.49 for retest at visit 3. CONCLUSIONS The Rett Syndrome Behavioural Questionnaire did not achieve acceptable standards as an outcome assessment for clinical trials in Rett syndrome. BACKGROUND Although behavioral therapy is an effective approach to reduce tics in children and adults, there is an insufficient availability and accessibility of behavioral therapy in the community. OBJECTIVE The goal of the study was to test the clinical efficacy of home-based, parent-provided behavioral therapy in children with Tourette syndrome aged seven to 13&nbsp;years. METHOD An instructional habit reversal training-based video and guide was developed for use by parents. Eligible families, in this 10-week study, were enrolled in either a home-based therapy (DVD) group (received disk and written instructions) or an in-person therapist group (had scheduled visits with the therapist). Outcome scales included the Yale Global Tic Severity Scale, both the total Tic Severity Score and total Global Severity Score, and the parent report of Clinical Global Impressions of Improvement. RESULTS Forty-four children (mean age&nbsp;=&nbsp;10.21&nbsp;±&nbsp;1.69&nbsp;years) were enrolled into either the DVD (n&nbsp;=&nbsp;33) or in-person therapist (n&nbsp;=&nbsp;11) groups.